MedDataX Logo

A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

NCT ID: NCT03912233

Last Updated: 2023-04-20

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

87 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-04-30

Study Completion Date

2019-12-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to evaluate the safety, tolerability and efficacy of VX-121 combination therapy in subjects with cystic fibrosis (CF).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis

NCT03768089

Cystic Fibrosis
COMPLETED PHASE1/PHASE2

A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

NCT03224351

Cystic Fibrosis
COMPLETED PHASE2

A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

NCT03911713

Cystic Fibrosis
COMPLETED PHASE2

A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)

NCT05033080

Cystic Fibrosis
COMPLETED PHASE3

A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic Fibrosis

NCT02951195

Cystic Fibrosis
COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Part 1: Placebo

Participants received placebo matched to VX-121/TEZ/VX-561 triple combination (TC) for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebos matched to VX-121, TEZ, and VX-561 for oral administration.

Part 1: VX-121/TEZ/VX-561 TC - Low Dose

Participants received VX-121 5 milligram (mg) once daily (qd)/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period

Group Type EXPERIMENTAL

VX-121

Intervention Type DRUG

Tablets for oral administration.

TEZ

Intervention Type DRUG

TEZ tablet for oral administration.

VX-561

Intervention Type DRUG

Tablets for oral administration.

Part 1: VX-121/TEZ/VX-561 TC - Medium Dose

Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.

Group Type EXPERIMENTAL

VX-121

Intervention Type DRUG

Tablets for oral administration.

TEZ

Intervention Type DRUG

TEZ tablet for oral administration.

VX-561

Intervention Type DRUG

Tablets for oral administration.

Part 1: VX-121/TEZ/VX-561 TC - High Dose

Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.

Group Type EXPERIMENTAL

VX-121

Intervention Type DRUG

Tablets for oral administration.

TEZ

Intervention Type DRUG

TEZ tablet for oral administration.

VX-561

Intervention Type DRUG

Tablets for oral administration.

Part 2: TEZ/IVA

Following run-in period with TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg/IVA 150 mg q12h for 4 weeks in the washout period.

Group Type ACTIVE_COMPARATOR

TEZ/IVA

Intervention Type DRUG

Fixed-dose combination tablets for oral administration.

IVA

Intervention Type DRUG

Tablets for oral administration.

Part 2: VX-121/TEZ/VX-561 TC - High Dose

Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.

Group Type EXPERIMENTAL

VX-121

Intervention Type DRUG

Tablets for oral administration.

TEZ

Intervention Type DRUG

TEZ tablet for oral administration.

VX-561

Intervention Type DRUG

Tablets for oral administration.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VX-121

Tablets for oral administration.

Intervention Type DRUG

TEZ

TEZ tablet for oral administration.

Intervention Type DRUG

VX-561

Tablets for oral administration.

Intervention Type DRUG

TEZ/IVA

Fixed-dose combination tablets for oral administration.

Intervention Type DRUG

IVA

Tablets for oral administration.

Intervention Type DRUG

Placebo

Placebos matched to VX-121, TEZ, and VX-561 for oral administration.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

VX-661 Tezacaftor CTP-656 Deutivacaftor (D-IVA) VX-661/VX-770 Tezacaftor/Ivacaftor VX-770 Ivacaftor

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Part 1: Heterozygous for F508del and an MF mutation (F/MF)
* Part 2: Homozygous for F508del (F/F)
* FEV1 value ≥40% and ≤90% of the predicted mean for age, sex, and height

Exclusion Criteria

* History of clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* History of solid organ or hematological transplantation
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Keck Medical Center of University of Southern California

Los Angeles, California, United States

Site Status

Kaiser Permanente

Oakland, California, United States

Site Status

University of Kentucky.

Lexington, Kentucky, United States

Site Status

Tulane Medical Center

New Orleans, Louisiana, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Santiago Reyes, M.D.

Oklahoma City, Oklahoma, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

Essen, , Germany

Site Status

Pneumologisches Studienzentrum Muenchen-West

München, , Germany

Site Status

Academic Medical Center

Amsterdam, , Netherlands

Site Status

University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

Heidelberglaan, , Netherlands

Site Status

UMC St. Radboud

Nijmegen, , Netherlands

Site Status

Erasmus Medical Center

Rotterdam, , Netherlands

Site Status

HagaZiekenhuis van den Haag

The Hague, , Netherlands

Site Status

Hospital de Santa Maria

Lisbon, , Portugal

Site Status

University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Site Status

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

London, , United Kingdom

Site Status

Wythenshawe Hospital

Manchester, , United Kingdom

Site Status

The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status

All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

Penarth, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Germany Netherlands Portugal United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Uluer AZ, MacGregor G, Azevedo P, Indihar V, Keating C, Mall MA, McKone EF, Ramsey BW, Rowe SM, Rubenstein RC, Taylor-Cousar JL, Tullis E, Yonker LM, Chu C, Lam AP, Nair N, Sosnay PR, Tian S, Van Goor F, Viswanathan L, Waltz D, Wang LT, Xi Y, Billings J, Horsley A; VX18-121-101; VX18-561-101 Study Groups. Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials. Lancet Respir Med. 2023 Jun;11(6):550-562. doi: 10.1016/S2213-2600(22)00504-5. Epub 2023 Feb 23.

Reference Type DERIVED
PMID: 36842446 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

VX18-121-101

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis
NCT02951182 COMPLETED PHASE2
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
NCT05422222 RECRUITING PHASE3
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
NCT05076149 COMPLETED PHASE3
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
NCT05444257 ACTIVE_NOT_RECRUITING PHASE3
A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03525444 COMPLETED PHASE3
Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
NCT03691779 COMPLETED PHASE3
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
NCT02709109 COMPLETED PHASE2
A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult Subjects
NCT03486236 COMPLETED PHASE1
A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
NCT03029455 COMPLETED PHASE1
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
NCT01531673 COMPLETED PHASE2
A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)
NCT05668741 RECRUITING PHASE1/PHASE2
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
NCT04043806 COMPLETED PHASE3
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
NCT03525574 COMPLETED PHASE3
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
NCT02516410 COMPLETED PHASE3
A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
NCT03525548 COMPLETED PHASE3
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
NCT02070744 COMPLETED PHASE2
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older
NCT05153317 ACTIVE_NOT_RECRUITING PHASE3
A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
NCT03227471 COMPLETED PHASE1/PHASE2
A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
NCT02347657 COMPLETED PHASE3
A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
NCT02392234 COMPLETED PHASE3
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)
NCT02953314 COMPLETED PHASE3
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
NCT04537793 COMPLETED PHASE3
Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
NCT00865904 COMPLETED PHASE2
A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
NCT02508207 COMPLETED PHASE2
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
NCT03447249 COMPLETED PHASE3